Biopharmaceuticals: large molecules, enormous potential
Biopharmaceutical drug products serve as the basis for many new therapeutic treatments and, at the same time, represent beacons of hope for numerous patients. They have the potential to specifically target previously untreatable diseases. However, the work on these diverse active ingredients, from research to production, is highly complex and requires special expertise as well as specific technologies and devices.
“Biopharmaceuticals are playing an ever greater part in treating a wide variety of diseases. A constant flow of groundbreaking innovations, such as immunotherapies and antibody-drug conjugates, has been entering the market. New treatment methods, such as gene, cell, and viral therapies, are currently in development or already on the market. The development these new therapies gives new hope to patients whose treatment options have so far been limited.”
Ralf Schumacher, Head of Development Biologicals at Boehringer Ingelheim
What is the potential of biopharmaceuticals?
So far, a wide variety of biopharmaceuticals have been used to treat serious diseases, such as autoimmune or rheumatic diseases, multiple sclerosis, or cancer, and have really made a difference. In the future, researchers in oncology are hoping to achieve further breakthroughs, such as through the targeted activation of the body's own immune system against the tumor. In addition, gene-modified cell therapies and oncolytic virus therapies are some of the options that offer great hope. Yet there is a multitude of possible applications. Biopharmaceuticals also offer promising treatment options for rare diseases for which no treatment options has existed yet or has not been very effective, such as rare skin diseases.
In 2022, almost half of all new drugs approved in Europe were biopharmaceuticals (46 percent)1, while in the USA the figure is over 40 percent2. In total, more than 350 biopharmaceutical products were approved for the German market in 2021. According to the current biotech report prepared by the German Association of Research-Based Pharmaceutical Companies (Verband Forschender Arzneimittelhersteller e.V.), the number of biopharmaceutical pipeline projects in Germany is continuing to increase.3
Biopharmaceuticals: what makes them special?
Biopharmaceuticals are complex proteins or peptides that are produced in living organisms using sophisticated biotechnological processes. While chemical pharmaceuticals have a comparably simple structure and can be replicated using standardized processes, biopharmaceuticals are produced in living cell cultures or microorganisms. The cells already contain the tools they need (e.g., yeasts or bacteria), but they need help to produce the desired active ingredient. In this process, the cells are genetically modified to yield the desired molecules from which the pharmaceutical product is manufactured.
Biopharmaceutical manufacturing process
The producing cells are very sensitive. The slightest change in their environment, such as fluctuations in physical parameters like temperature, pH value, or pressure, can significantly affect the production of the desired molecule. Developing a stable production process is therefore especially critical as it affects the final product quality. Continuous process controls are thus essential.
The majority of approved biopharmaceuticals are monoclonal antibodies, which can be used in a highly targeted way. However, biopharmaceuticals also include modified proteins, as well as vaccines, peptides, gene therapies, and cell therapies.
What makes this type of drug substance special is its size. They really are tiny giants. While chemical molecules have a small mass, acetylsalicylic acid (aspirin), for example, has a mass of approximately 0.18 kilodaltons, biopharmaceutical molecules quickly reach 10,000 to 100,000 times that size.
Chemical molecules vs. biopharmaceuticals
Boehringer Ingelheim is a pioneer in biopharmaceutical research and has been active in this field since 1963. In recent years, we have received four approvals in this area based on our own research and development work. Moreover, under the brand name Boehringer Ingelheim BioXcellence™ the company is a world leader in contract manufacturing of biopharmaceuticals.
1 Biotech-Report „Medizinische Biotechnologie in Deutschland 2022“, Boston Consulting Group, vfa bio – Verband Forschender Arzneimittelhersteller e.V.
2 Mullard, A. (2023). 2022 FDA approvals. Nature Reviews Drug Discovery, 22(2), 83–88. https://doi.org/10.1038/d41573-023-00001-3
3 Biotech-Report „Medizinische Biotechnologie in Deutschland 2022“, Boston Consulting Group, vfa bio – Verband Forschender Arzneimittelhersteller e.V.